#BigPharma making presence felt in the #raredisease world

Once a neglected market of the pharmaceutical industry, orphan drugs are now very much in favour as large drug makers search for revenue to cover a decline in sales of blockbuster treatments losing patent protection.

GSK and Pfizer have established focused rare disease research units and are targeting ADA-SCID and Gaucher disease respectively.

Collaboration has become key for big pharma as they move into the orphan drug space and are increasingly looking towards orphan drug specialists who have long been pioneering to treat true unmet medical needs.

Protalix Biotherapeutics are in partnership with Pfizer in the search for a treatment for Gaucher disease, and data has recently been resubmitted to the FDA for market approval. Amicus Therapeutics, headed by their inspirational CEO John Crowley are in alliance with GSK and boast a strong candidate in phase III clinical trials for the treatment of Fabry disease.